PhRMA Statement on Clinical Trials and Bad Pharma

“Dr. Ben Goldacre’s book Bad Pharma provides a one-sided and factually questionable view of the clinical trial process led by the innovative biopharmaceutical sector. The book ignores that the current clinical trial system, which is essential to the development of new medicines that save and improve lives, is scientifically rigorous, tightly regulated and working well.

“The book’s unfounded and sensational claims may attract attention, but they do not advance biomedical science or patient health. Based on cherry-picked examples and incomplete stories, the accusations leave countless unanswered questions that could threaten public health and the development of future medicines. Criticizing the research process without acknowledging the vigorous oversight and broad commitment to safe and ethical conduct is a disservice to the tens of thousands of scientists exploring new medicines and the millions of patients hoping for cures.

“New medicines are the result of careful collaboration among sponsoring companies, regulatory agencies, investigative site staff, medical professionals, hospitals, academia, institutional review boards and ethics committees. The Food and Drug Administration (FDA), along with international standards such as the Guideline for Good Clinical Practice of the International Conference on Harmonization, govern the safe and ethical conduct of clinical trials. Additionally, PhRMA member companies agree to the organization’s Principles on Conduct of Clinical Trials and Communication of Clinical Trial Results, which were updated in 2009. The clinical trial process is the safest and most productive it has been in history.

“Clinical trials conducted by the biopharmaceutical sector have led to significant progress against scores of diseases. Medicines tested through the clinical trial process have contributed to dramatically reduced death rates (including a 79 percent reduction in deaths from HIV/AIDS, a 30 percent decrease in deaths from heart disease and a 20 percent decrease in deaths from cancers).

“The demands by Dr. Goldacre and the British Medical Journal (BMJ) to release patient-level clinical trial data are irresponsible with potentially harmful consequences for future medicine development. The recommendations would jeopardize patient privacy and could serve as a deterrent to individuals considering participation in trials. It would also encourage second-guessing of the regulatory approval process, which would be disastrous for patients. The FDA has the most advanced and rigorous review process for potential new medicines and it is continuously improving its regulatory and scientific capabilities.

“Dr. Goldacre often cites a January 2013 study published in the British Medical Journal that purported to evaluate whether trial results were present on ClinicalTrials.gov for records that the authors deemed to require results reporting under FDAAA. The authors claimed that only 22% of these trials had reported results within one year of completion of the trial. However, a preliminary analysis by the FDA presented at The Thirteenth Annual Pharmaceutical Regulatory and Compliance Congress in November 2012 found that the BMJ study did not account accurately for all elements of the statutory definition for “applicable clinical trials,” that should be posted. The FDA analysis of posted clinical trial data from May 2012 appears to contradict the BMJ study. FDA found that the vast majority (78%) of trials that met the criteria for required reporting were posted on ClinicalTrials.gov and only a low number appeared to be missing. 31% of trials that the BMJ study included as “missing” were classified by the FDA as not required to be submitted. While 21% of trials not reported is significant and the causes of underreporting must be studied and remedied, the BMJ study dramatically overstates the problem and does not reflect the full scale of clinical trial data sharing. Biopharmaceutical companies welcome an informed discussion of possible noncompliance in the reporting system with FDA, NIH and other stakeholders.

“The current clinical trial system allows for the sharing of trial results to government regulators, the academic and medical communities, physicians and the public through submissions to the FDA, presentations at medical conferences, publication in peer-reviewed journals and registration on ClinicalTrials.gov, the clearinghouse of clinical trials maintained by the National Institutes of Health. As of February 4 2013, ClinicalTrials.gov reported 139,847 studies in all 50 states and in 182 countries. This is an exponential increase from 2005, when there were approximately 12,000 trials reported. The biopharmaceutical sector is among the most transparent research-intensive industries; few others publish the research findings that power their products.

“Following FDA approval of a medicine, the FDA releases a comprehensive summary approval package, which includes analysis of all significant clinical trials used for the approval process. FDA constantly monitors approved medicines and updates approved labeling accordingly. The approved labeling contains the synthesis of all known clinical data about a medicine. The Drugs@FDA database enables searches for information about FDA-approved brand name and generic medicines and therapeutic biological products, including labels, patient information, approval letters, and other information.

“Voluntary participation in clinical trials provides opportunities for patients to gain access to a new treatment (sometimes the only option for their condition), obtain high-quality care in leading medical facilities, play a more active role in their health care and contribute to medical research. Biopharmaceutical companies recognize the important value of these patients in the development of new medicines, and are committed to protecting their privacy.

“Medical innovation would not be possible without the commitment to research and development by the American biopharmaceutical sector. Since 2000, PhRMA member companies have invested more than $500 billion in R&D. According to the National Science Foundation, nearly 20 percent of all domestic R&D can be attributed to the biopharmaceutical sector – the single largest share of all U.S. business R&D.

“There are currently over 5,000 new medicines in the pipeline, 70 percent of which are potential first-in-class medicines that could provide exciting new approaches to treating diseases for patients. Personalized medicines and drugs for rare diseases account for a growing share of the research being conducted by the biopharmaceutical sector.

“Clinical trials are critical to fulfilling the promise of this robust pipeline. Our sector and other stakeholders in the trials process welcome a constructive dialogue and are focused on protecting the privacy and safety of study participants, overcoming barriers to greater participation and discovery. Debating the self-serving claims of Dr. Goldacre does nothing to advance patient health.”

About PhRMA

The Pharmaceutical Research and Manufacturers of America (PhRMA) represents the country’s leading innovative biopharmaceutical research and biotechnology companies, which are devoted to discovering and developing medicines that enable patients to live longer, healthier, and more productive lives. Since 2000, PhRMA member companies have invested over $500 billion in the search for new treatments and cures, including an estimated $49.5 billion in 2011 alone.http://www.phrma.org/